Lieberman, Chair of the Department of Psychiatry with extensive experience in experimental therapeutics, added: “Identifying susceptibility gene products is currently the best approach to identifying novel biologic targets and developing innovative psychopharmacologic treatments”.Ĭarlos Buesa, Oryzon’s CEO, said: "We are pleased to initiate this ground-breaking collaboration with researchers at the internationally renowned Columbia University. The emergence of this kind of "precise treatments" is an exciting moment in the battle against psychiatric disorders.” Dr. Gogos, Professor at Columbia University, said: “Following the enormous success in human psychiatric genetics, the goal of using knowledge from genetic studies to identify drugs that target specific biological disease mechanisms has begun to be realized. Markx, principal investigator of the study, said: “We are very excited to partner with Oyrzon in carrying out a baseline neuropsychiatric characterization study that will pave the way for one of the first precision psychiatry clinical trials involving vadifemstat for SETD1A-associated psychiatric disorders.” Dr. De novo mutations in other subunits comprising Set/COMPASS have also been reported in subjects with SCZ and ASD.ĭr. Rare variants in SETD1A have been demonstrated in large, unbiased studies to be associated with increased risk for schizophrenia and neurodevelopmental disorders with cognitive impairment. SETD1A encodes a catalytic subunit of the histone methyltransferase protein complex, named Set/COMPASS. In the field of precision psychiatry, vafidemstat is also being explored in Europe in patients with Phelan-McDermid syndrome – a variety of autism – who harbor a mutation in SHANK3. Vafidemstat is being currently explored in two Phase IIb clinical trials, one in BPD and the other in severe Covid-19 patients. Vafidemstat is an LSD1 inhibitor in Phase II clinical development that has shown a very good safety profile and has been shown to be effective in reducing agitation and aggression in clinical studies in patients with Alzheimer's disease, Borderline Personality Disorder (BPD), Attention-Deficit/Hyperactivity Disorder (ADHD) and Autism Spectrum Disorder (ASD). This study will inform the potential actionability of the SETD1A regulator protein by inhibition of LSD1 and the best endpoints for a future clinical study with vafidemstat. Markx, who is Professor of Psychiatry at Columbia University and the leader of the Columbia Precision Psychiatry group, will conduct a pilot study to characterize clinical profile of these individuals to determine their different degrees of cognitive impairment. Patients harboring inherited mutations in SETD1A have been identified in the Amish founder population in Pennsylvania. This finding opens the door for investigation of intervention with vafidemstat during a therapeutic window in adolescent and adult subjects. This work specifically demonstrated that these treatment effects occur in adult animals suggesting that deficiency of Setd1a function during early developmental stages does not irreversibly compromise the function of diseaserelated neural circuits.
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Critically, administration of an Oryzon’s LSD1 inhibitor produced a full rescue of both the behavioral and neuronal disease phenotypes. Gogos’ laboratory at Columbia University has shown that heterozygous loss-of-function (LOF) mutation of the Setd1a gene in mice results in alterations in axonal branching and cortical synaptic dynamics, accompanied by specific deficits in working memory that recapitulate human SCZ-related cognitive and behavioral deficits.
#Sander markx lab trial
The second is to perform exhaustive functional psychometric characterization of individuals carrying mutations in the SETD1A gene to build a foundation for a subsequent precision psychiatry clinical trial with vafidemstat for SETD1A-associated psychiatric disorders. This project has two parts: the first is to further characterize the therapeutic actionability of the SETD1A regulator protein encoded by this gene with LSD1 inhibitors at the molecular level in preclinical Setd1a models. SETD1A is a histone methyltransferase that is a key SCZ susceptibility gene. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with a strong unmet medical need, announced today the start of a precision medicine collaboration in schizophrenia (SCZ) with researchers from Columbia University in New York, Dr. MADRID, SPAIN and CAMBRIDGE, MA, UNITED STATES, December 17th, 2020 - Oryzon Genomics, S.A.
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